Not logged in Share Save
Natural history of limb girdle muscular dystrophy R1 (LGMDR1): a GRASP consortium study Hunn, Stephanie M.; Findlay, Andrew R.; Alfano, Lindsay N.; Jones, Aileen; Butler, Amanda; Lowes, Linda P.; Iammarino, Megan A.; Reash, Natalie F.; Pietruszewski, Lindsay; Sasidharan, Sandhya; Currence, Melissa; Statland, Jeffrey M.; Strahler, Talia; Will, Robert; Wicklund, Matthew; Dixon, Stacy; Augsburger, Renee; Mozaffar, Tahseen; Laubscher, Katie M.; Mockler, Shelley R. H.; Mathews, Katherine D.; Stinson, Nikia; Leung, Doris G.; Stark, Molly M.; Horton, Rebecca A.; Kang, Peter B.; James, Meredith K.; Clause, Amanda; Weihl, Conrad C.; Johnson, Nicholas E. Share Save
Share Save
Motor Function in Limb-Girdle Muscular Dystrophy R1/2A James, Meredith K.; Iammarino, Megan A.; Reash, Natalie F.; Steiner, Christopher L.; Knight, Audrey B.; Smith, Melissa A.; Moat, Dionne; Michell-Sodhi, Jassi; Wong, Karen S. W.; Grover, Emma; Robinson, Emma-Jayne; Mayhew, Anna G.; Eagle, Michelle; Elseed, Maha; Guglieri, Michela; Straub, Volker; Marini-Bettolo, Chiara; Muni-Lofra, Robert; Diaz-Manera, Jordi; Lowes, Linda Pax; Alfano, Lindsay N. Share Save
Share Save
Share Save
Clinical Trial Readiness in Limb Girdle Muscular Dystrophy R1 (LGMDR1): A GRASP Consortium Study Hunn, Stephanie M.; Alfano, Lindsay N.; Jones, Aileen; Butler, Amanda; Lowes, Linda P.; Iammarino, Megan A.; Reash, Natalie F.; Pietruszewski, Lindsay; Sasidharan, Sandhya; Currence, Melissa; Statland, Jeffrey M.; Strahler, Talia; Will, Robert; Wicklund, Matthew; Dixon, Stacy; Augsburger, Renee; Mozaffar, Tahseen; Laubscher, Katie M.; Mockler, Shelley R. H.; Mathews, Katherine D.; Stinson, Nikia; Leung, Doris G.; Stark, Molly M.; Horton, Rebecca A.; Kang, Peter B.; James, Meredith K.; Clause, Amanda; Weihl, Conrad C.; Johnson, Nicholas E. Share Save
Validity and Reliability of Clinical and Patient-Reported Outcomes in Multisystem Proteinopathy 1 Alfano, Lindsay N.; Iammarino, Megan A.; Reash, Natalie F.; Lowes, Linda P.; Pietruszewski, Lindsay; Adderley, Kathleen; Humphrey, Lauren; Knight, Audrey B.; Steiner, Christopher L.; Smith, Melissa A.; Sahenk, Zarife; Connolly, Anne M.; Almomen, Momen; D'Ambrosio, Eleonora S.; Peck, Nathan; Peck, Allison Share Save
Share Save
Prospective observational study of FKRP-related limb-girdle muscular dystrophy R9: A GRASP consortium study Alfano, Lindsay N.; James, Meredith K.; Petersen, Kristine v; Rudolf, Karen; Vissing, John; Augsburger, Renee; Mozaffar, Tahseen; Jones, Aileen; Butler, Amanda; Laubscher, Katie M.; Mockler, Shelley R. H.; Mathews, Katherine D.; Iammarino, Megan A.; Reash, Natalie F.; Pietruszewski, Lindsay; Lowes, Linda P.; Strahler, Talia; Wicklund, Matthew; Hunn, Stephanie; Weihl, Conrad C.; Sasidharan, Sandhya; Currence, Melissa; Statland, Jeffrey M.; Stinson, Nikia; Holzer, Megan; Leung, Doris G.; Lott, Donovan J.; Kang, Peter B.; Holsten, Scott; Desai, Urvi; Johnson, Nicholas E.; GRASP LGMD Consortium Share Save
Gross motor delays in infants and young boys with Duchenne muscular dystrophy Lowes, Linda P.; Reash, Natalie F.; Iammarino, Megan A.; Connolly, Anne M.; Pietruszewski, Lindsay; Smith, Melissa A.; Peng, Jing; Steiner, Christopher L.; Tsao, Chang-Yong; Waldrop, Megan A.; Flanigan, Kevin M.; Chagat, Shannon; Meyer, Alayne P.; Mendell, Jerry R.; Alfano, Lindsay N. Share Save
Share Save
Increase in Full-Length Dystrophin by Exon Skipping in Duchenne Muscular Dystrophy Patients with Single Exon Duplications: An Open-label Study Nicolau, Stefan; Malhotra, Jyoti; Kaler, Maryann; Coxen, Pamela Magistrado; Iammarino, Megan A.; Reash, Natalie F.; Frair, Emma C.; Wijeratne, Saranga; Kelly, Benjamin J.; White, Peter; Lowes, Linda P.; Waldrop, Megan A.; Flanigan, Kevin M. Share Save
Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results Mendell, Jerry R.; Pozsgai, Eric R.; Lewis, Sarah; Griffin, Danielle A.; Lowes, Linda P.; Alfano, Lindsay N.; Lehman, Kelly J.; Church, Kathleen; Reash, Natalie F.; Iammarino, Megan A.; Sabo, Brenna; Potter, Rachael; Neuhaus, Sarah; Li, Xiaoxi; Stevenson, Herb; Rodino-Klapac, Louise R. Share Save
WiTNNess: An international natural history study of infantile-onset TNNT1 myopathy Strauss, Kevin A.; Carson, Vincent J.; Bolettieri, Emilienne; Everett, Mariah; Bollinger, Ashton; Bowser, Lauren E.; Beiler, Keturah; Young, Millie; Edvardson, Simon; Fraenkel, Nitay; D'Amico, Adele; Bertini, Enrico; Lingappa, Lokesh; Chowdhury, Devyani; Lowes, Linda P.; Iammarino, Megan; Alfano, Lindsay N.; Brigatti, Karlla W. Share Save
Twelve-month functional change in Limb Girdle Muscular Dystrophy R9 / 2I Johnson, Nicholas; Bates, Kameron; Xie, Han; Alfano, Lindsay; Vissing, John; Mozaffar, Tahseen; Mathews, Katherine; Lowes, Linda; Wicklund, Matthew; Weihl, Conrad; Leung, Doris; Kang, Peter; Zingariello, Carla; Desai, Urvi; DeSpain, Erin; Romain, Mary St.; Statland, Jeffrey Share Save
Long-term Safety and Efficacy in Patients with DMD 4 Years Post-Treatment with Delandistrogene Moxeparvovec (SRP-9001) in a Phase 1/2a Study Mendell, Jerry; Sahenk, Zarife; Lehman, Kelly; Lowes, Linda; Reash, Natalie; Iammarino, Megan; Alfano, Lindsay; Lewis, Sarah; Church, Kathleen; Shell, Richard; Potter, Rachael; Griffin, Danielle; Hogan, Mark; Wang, Shufang; Mason, Stefanie; Darton, Eddie; Rodino-Klapac, Louise Share Save
A Phase 2 Clinical Trial Evaluating the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Patients with Duchenne Muscular Dystrophy (DMD) Mendell, Jerry; Shieh, Perry; Sahenk, Zarife; Lehman, Kelly; Lowes, Linda; Reash, Natalie; Iammarino, Megan; Alfano, Lindsay; Sabo, Brenna; Woods, Jeremy; Skura, Christy; Mao, Howard; Staudt, Loretta; Potter, Rachael; Griffin, Danielle; Lewis, Sarah; Wang, Shufang; Singh, Tejdip; Rodino-Klapac, Louise Share Save
Toward patient-centered treatment goals for duchenne muscular dystrophy: insights from the Your Voice study Schwartz, Carolyn E.; Jackson, Skyler; Valentine, James; Miller, Natalie; Lowes, Linda; Edwards, Danielle; McSherry, Christine; Savva, Dimitrios; Lowe, Alex; McSherry, Jordan; Engel, Patti Share Save
Validation of the North Star Assessment for Limb-Girdle Type Muscular Dystrophies James, Meredith K.; Alfano, Lindsay N.; Muni-Lofra, Robert; Reash, Natalie F.; Sodhi, Jassi; Iammarino, Megan A.; Moat, Dionne; Shannon, Kianna; McCallum, Michelle; Richardson, Mark; Eagle, Michelle; Straub, Volker; Marini-Bettolo, Chiara; Lowes, Linda P.; Mayhew, Anna G. Share Save
.png){kind=icon}
.png){kind=icon}
.png){kind=icon}
.png){kind=icon}
.png){kind=icon}
Share.png){kind=icon}
Save
Not logged in.png){kind=icon}